Diamond-Blackfan anemia (DBA) is a congenital hypoproliferative anemia that generally presents in infancy. The mainstays of treatment, prednisone and transfusion therapy, have long-term toxicity in many patients, and bone marrow transplantation with an HLA-matched donor is an option for only a minority of patients. Most importantly, patients with DBA have an increased risk of progression of myelodysplastic syndrome, leukemia, and aplastic anemia compared to the general population.